On 21 March 2012, orphan designation (EU/3/12/973) was granted by the European Commission to NDA Regulatory Science Ltd, United Kingdom, for recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome).

The sponsorship was transferred to NDA Group AB, Sweden, in December 2013 and subsequently to Ultragenyx UK Limited, United Kingdom, in May 2015.

Recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type VII (Sly syndrome) has been authorised in the EU as Mepsevii since 23 August 2018.

This medicine is now known as vestronidase alfa.

Key facts

Active substance
Recombinant human beta-glucuronidase (vestronidase alfa)
Medicine name
Disease / condition
Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Mepsevii at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF iconorphan medicine assessment report .

Sponsor's contact details

Ultragenyx Germany GmbH
Friedrichstrasse 191
10117 Berlin
Tel. +49 30 2065 9217

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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